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(11)

EP 3 976 202 A1

(12)

(43) Date of publication: 06.04.2022 Bulletin 2022/14 (21) Application number: 20730007.0 (22) Date of filing: 28.05.2020

(51) International Patent Classification (IPC):  A61P 37/02(2006.01) A61K 48/00(2006.01) A61K 38/00(2006.01) (52) Cooperative Patent Classification (CPC): A61K 38/00; A61P 37/02; A61K 35/17; C07K 14/55; C07K 14/5418; C07K 14/5443; C07K 14/7051; C07K 2319/03; A61P 35/00; A61P 31/20; A61K 39/39; C12N 2730/10134; A61K 39/12; A61K 2039/5158; A61K 39/0005; A61K 2039/6056; C07K 16/246; C07K 16/2854; C07K 2317/76; C12N 15/86; A01K 2227/105; A01K 2267/0337; A61K 48/005; A61K 48/0058; C12N 2830/008; C12N 2740/16043; C12N 2730/10122   C-Sets: A61K 35/17, A61K 2300/00; (86) International application number: PCT/EP2020/064933 (87) International publication number: WO 2020/239964 (03.12.2020 Gazette 2020/49)

(84) Designated Contracting States: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA ME Designated Validation States: KH MA MD TN (30) Priority: 28.05.2019 GB 201907493 (71) Applicants: Ospedale San Raffaele S.r.l. 20132 Milano (MI) (IT) Fondazione Telethon 00185 Rome (IT)	(72) Inventors: NALDINI, Luigi 20132 Milan (IT) CANTORE, Alessio 20132 Milan (IT) IANNACONE, Matteo 20132 Milan (IT) GUIDOTTI, Luca 20132 Milan (IT) OSTUNI, Renato 20132 Milan (IT) (74) Representative: D Young & Co LLP  120 Holborn London EC1N 2DY London EC1N 2DY (GB)